AlloSCT may offer better prevention of silent cerebral infarcts and improved quality of life in patients with sickle cell disease (SCD). Adjuvant zinc sulfate and ursodeoxycholic acid may reduce ...

The incidence of myasthenia gravis (MG) is increasing in the Czech Republic, according to a new study by Czech scientists.

Patients underwent follow-up evaluations on days 4, 10, and 21 post-RS, followed by monthly assessments extending u to 6 months. It is well known that individuals with hemophilia are at an elevated ...

This case underscores the importance of individualized treatment plans and the need for meticulous perioperative care in patients with bleeding disorders. Among individuals with hemophilia, ...

There were no serious adverse events in children who underwent sessions of robot-assisted gait therapy with the device. ATLAS 2030, the medical lower-body exoskeleton for children is safe for patients ...

Sotatercept at 0.7 mg/kg subcutaneous every 3 weeks optimally balanced efficacy and safety for those with PAH. Sotatercept, a practice-changing therapy for pulmonary arterial hypertension (PAH), ...

Avapritinib results in better outcomes among patients with advanced systemic mastocytosis (SM) compared with other therapies, according to new research.

Sandy Siegel, founder and president of the Siegel Rare Neuroimmune Association (Photo by Larry Luxner) Anthropologist Sandy Siegel launched the group after his late wife was diagnosed with transverse ...

Rosalind Kalb is lead senior programs consultant for Can-Do MS, a Colorado-based nonprofit health and wellness organization. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...

Steve Van Wormer is cofounder and director of the Phaware Global Association, a nonprofit group that raises awareness of pulmonary hypertension (Photo by Larry Luxner) I’m Aware That I’m Rare has had ...

As of November 2024, approximately 137 clinical trials for spinal muscular atrophy (SMA) are ongoing or pending: 8 trials that are not yet recruiting, 74 trials that are currently recruiting, 26 ...

As of November 2024, approximately 156 clinical trials for Duchenne muscular dystrophy (DMD) are ongoing or pending: 16 trials not yet recruiting, 65 currently recruiting, 28 trials that are active ...